Trusted updates, explained simply
This study looked at how people with Rett syndrome in the United States used trofinetide, an FDA-approved treatment, over time.
🔬 Journal of medical economics
This genomic study explored the genetic roots of hair pulling (trichotillomania) and skin picking (excoriation disorder) in families, finding connections to genes involved in obsessive-compulsive disorder.
🔬 Translational psychiatry
A study compared how children with cochlear implants, children with normal hearing, and normal hearing children listening through a 'vocoder' device perceived and produced speech sounds, finding similar overall error rates but different patterns for specific sounds.
🔬 Ear and hearing
A study found that only about one-quarter of eligible individuals with Rett Syndrome received the newly approved medication, Trofinetide, shortly after its approval in the United States.
🔬 Journal of health economics and outcomes research
Researchers have finished giving the study treatment to all 25 children in a major trial for a new gene therapy called NGN-401.
🔬 ClinicalTrials.gov
A new clinical study is checking how many children have antibodies that might block gene therapy from working.
🔬 ClinicalTrials.gov
Scientists recently gathered to share new findings on brain health, memory, and aging in individuals with Down Syndrome.
🔬 PubMed (Neuromolecular Medicine)
This study investigated how the brains of children and teenagers with autism process visual attention, finding specific differences in brain activity when focusing on the right side compared to typically developing peers.
🔬 bioRxiv : the preprint server for biology
A new clinical trial is recruiting teenagers with Down syndrome to test a medicine that may help with memory and focus.
🔬 ClinicalTrials.gov
A new phase 2 clinical trial is testing a targeted medication for children with Dravet Syndrome and other SCN1A-related conditions.
🔬 ClinicalTrials.gov
The FDA recently hosted a workshop to find ways to get gene therapy into pediatric clinical trials sooner and more safely.
🔬 U.S. Food and Drug Administration (FDA)
Researchers found that a new antisense therapy can fix brain signaling issues in lab models of autism caused by SHANK mutations.
🔬 Nature Communications
Researchers found that the size of certain parts of the brain's outer layer may be linked to how well children with ADHD perform in school.
🔬 PubMed (National Library of Medicine)
A recent review explains how sleep disturbances are common in Rett Syndrome and how they relate to a child's mood and behavior during the day.
🔬 PubMed
New research suggests that children diagnosed with ADHD may have a higher chance of certain physical health challenges as they grow into adults.
🔬 JAMA Network Open
A large-scale study has identified new areas in human DNA that may be responsible for ADHD traits, providing a roadmap for future gene-based therapies.
🔬 Nature Genetics
A 'one-shot' viral gene therapy has been developed to restore a critical protein needed for brain development in MEF2C-related disorders.
🔬 National Institutes of Health (NIH)
An international clinical trial is currently testing a new oral medication designed to help young people with Down Syndrome manage daily tasks and memory.
🔬 ClinicalTrials.gov
The first clinical trial for a gene therapy targeting the SHANK3 gene is showing early signs of safety and benefit in young children.
🔬 ClinicalTrials.gov
A new review examines the safety of using viral vectors—tools in gene therapy—to address brain-related conditions like ADHD.
🔬 NIH (National Institutes of Health)
This study is comparing two different medications, intranasal dexmedetomidine and oral midazolam, to see which works better and is safer for reducing anxiety in children aged 2-10 before elective surgery.
🔬 Saglik Bilimleri Universitesi Gazi Yasargil Training and Research Hospital
The World Health Organization (WHO) has released a new training program to help caregivers of children with autism manage stress and provide effective home support.
🔬 World Health Organization
A new technique described in 'Nature' allows for the safe insertion of large pieces of DNA into the genome, which could help deliver gene therapies for Down syndrome.
🔬 Nature
A study found that babies frequently use both single words and physical actions or gestures to communicate, and mothers are more likely to respond when these actions clearly match what the baby is saying.
🔬 Infancy : the official journal of the International Society on Infant Studies
A recent review for doctors highlights how new gene-based therapies are changing the way Rett Syndrome is managed.
🔬 PubMed (NIH)
A 2024 expert review offers practical guidance for doctors and families on managing common digestive issues in Rett syndrome, including side effects from the newly approved medication trofinetide.
🔬 Expert Review of Gastroenterology & Hepatology
This study aims to determine if an intervention supporting caregivers in using educational media with children can improve early literacy skills, comparing it to shared book reading and no intervention.
🔬 ClinicalTrials.gov
This project aims to create a UK-wide research network to improve the understanding and treatment of eating disorders by connecting child and adult services for better research collaboration.
🔬 clinicaltrials.gov
A new pilot study is testing whether online training for doctors and therapists can improve how they use 'gait analysis' to help children walk better.
🔬 PubMed
This study plans to investigate if giving women an intranasal medication called dexmedetomidine before labor pain relief can help prevent postpartum depression after vaginal delivery.
🔬 Chengdu Jinjiang Maternity and Child Health Hospital
A 2023 peer-reviewed study used data from the US Rett Syndrome Natural History Study to identify the top concerns reported by caregivers of people with Rett syndrome and related disorders.
🔬 Journal of Neurodevelopmental Disorders
In March 2023, the FDA approved Daybue (trofinetide) as the first-ever treatment for Rett syndrome in adults and children 2 years and older. A clinical trial showed statistically significant improvement compared to placebo.
🔬 U.S. Food and Drug Administration (FDA)
A Phase 1/2/3 clinical trial is testing a one-time gene therapy called TSHA-102, delivered by spinal injection, in females ages 6 to under 22 with classic Rett syndrome.
🔬 ClinicalTrials.gov — Taysha Gene Therapies, Inc.
A large group of medical experts created the first consensus-based care guidelines for Rett syndrome, covering the full lifespan from childhood through adulthood.
🔬 BMJ Paediatrics Open
A 2019 review study found that children with Rett syndrome may show subtle signs of the condition before the well-known regression at age 2, suggesting there may be an earlier window for diagnosis and support.
🔬 Neuroscience and Biobehavioral Reviews
This completed study investigated how factors like family functioning, coping methods, and perceived illness burden impact caregivers of individuals with Rett Syndrome.
🔬 National Human Genome Research Institute (NHGRI)
This research digest is for educational and community support purposes only. It is not medical advice, diagnosis, or treatment. Always speak with a qualified doctor, therapist, or medical professional before making decisions about care.
Trusted updates, explained simply
This study looked at how people with Rett syndrome in the United States used trofinetide, an FDA-approved treatment, over time.
🔬 Journal of medical economics
This genomic study explored the genetic roots of hair pulling (trichotillomania) and skin picking (excoriation disorder) in families, finding connections to genes involved in obsessive-compulsive disorder.
🔬 Translational psychiatry
A study compared how children with cochlear implants, children with normal hearing, and normal hearing children listening through a 'vocoder' device perceived and produced speech sounds, finding similar overall error rates but different patterns for specific sounds.
🔬 Ear and hearing
A study found that only about one-quarter of eligible individuals with Rett Syndrome received the newly approved medication, Trofinetide, shortly after its approval in the United States.
🔬 Journal of health economics and outcomes research
Researchers have finished giving the study treatment to all 25 children in a major trial for a new gene therapy called NGN-401.
🔬 ClinicalTrials.gov
A new clinical study is checking how many children have antibodies that might block gene therapy from working.
🔬 ClinicalTrials.gov
Scientists recently gathered to share new findings on brain health, memory, and aging in individuals with Down Syndrome.
🔬 PubMed (Neuromolecular Medicine)
This study investigated how the brains of children and teenagers with autism process visual attention, finding specific differences in brain activity when focusing on the right side compared to typically developing peers.
🔬 bioRxiv : the preprint server for biology
A new clinical trial is recruiting teenagers with Down syndrome to test a medicine that may help with memory and focus.
🔬 ClinicalTrials.gov
A new phase 2 clinical trial is testing a targeted medication for children with Dravet Syndrome and other SCN1A-related conditions.
🔬 ClinicalTrials.gov
The FDA recently hosted a workshop to find ways to get gene therapy into pediatric clinical trials sooner and more safely.
🔬 U.S. Food and Drug Administration (FDA)
Researchers found that a new antisense therapy can fix brain signaling issues in lab models of autism caused by SHANK mutations.
🔬 Nature Communications
Researchers found that the size of certain parts of the brain's outer layer may be linked to how well children with ADHD perform in school.
🔬 PubMed (National Library of Medicine)
A recent review explains how sleep disturbances are common in Rett Syndrome and how they relate to a child's mood and behavior during the day.
🔬 PubMed
New research suggests that children diagnosed with ADHD may have a higher chance of certain physical health challenges as they grow into adults.
🔬 JAMA Network Open
A large-scale study has identified new areas in human DNA that may be responsible for ADHD traits, providing a roadmap for future gene-based therapies.
🔬 Nature Genetics
A 'one-shot' viral gene therapy has been developed to restore a critical protein needed for brain development in MEF2C-related disorders.
🔬 National Institutes of Health (NIH)
An international clinical trial is currently testing a new oral medication designed to help young people with Down Syndrome manage daily tasks and memory.
🔬 ClinicalTrials.gov
The first clinical trial for a gene therapy targeting the SHANK3 gene is showing early signs of safety and benefit in young children.
🔬 ClinicalTrials.gov
A new review examines the safety of using viral vectors—tools in gene therapy—to address brain-related conditions like ADHD.
🔬 NIH (National Institutes of Health)
This study is comparing two different medications, intranasal dexmedetomidine and oral midazolam, to see which works better and is safer for reducing anxiety in children aged 2-10 before elective surgery.
🔬 Saglik Bilimleri Universitesi Gazi Yasargil Training and Research Hospital
The World Health Organization (WHO) has released a new training program to help caregivers of children with autism manage stress and provide effective home support.
🔬 World Health Organization
A new technique described in 'Nature' allows for the safe insertion of large pieces of DNA into the genome, which could help deliver gene therapies for Down syndrome.
🔬 Nature
A study found that babies frequently use both single words and physical actions or gestures to communicate, and mothers are more likely to respond when these actions clearly match what the baby is saying.
🔬 Infancy : the official journal of the International Society on Infant Studies
A recent review for doctors highlights how new gene-based therapies are changing the way Rett Syndrome is managed.
🔬 PubMed (NIH)
A 2024 expert review offers practical guidance for doctors and families on managing common digestive issues in Rett syndrome, including side effects from the newly approved medication trofinetide.
🔬 Expert Review of Gastroenterology & Hepatology
This study aims to determine if an intervention supporting caregivers in using educational media with children can improve early literacy skills, comparing it to shared book reading and no intervention.
🔬 ClinicalTrials.gov
This project aims to create a UK-wide research network to improve the understanding and treatment of eating disorders by connecting child and adult services for better research collaboration.
🔬 clinicaltrials.gov
A new pilot study is testing whether online training for doctors and therapists can improve how they use 'gait analysis' to help children walk better.
🔬 PubMed
This study plans to investigate if giving women an intranasal medication called dexmedetomidine before labor pain relief can help prevent postpartum depression after vaginal delivery.
🔬 Chengdu Jinjiang Maternity and Child Health Hospital
A 2023 peer-reviewed study used data from the US Rett Syndrome Natural History Study to identify the top concerns reported by caregivers of people with Rett syndrome and related disorders.
🔬 Journal of Neurodevelopmental Disorders
In March 2023, the FDA approved Daybue (trofinetide) as the first-ever treatment for Rett syndrome in adults and children 2 years and older. A clinical trial showed statistically significant improvement compared to placebo.
🔬 U.S. Food and Drug Administration (FDA)
A Phase 1/2/3 clinical trial is testing a one-time gene therapy called TSHA-102, delivered by spinal injection, in females ages 6 to under 22 with classic Rett syndrome.
🔬 ClinicalTrials.gov — Taysha Gene Therapies, Inc.
A large group of medical experts created the first consensus-based care guidelines for Rett syndrome, covering the full lifespan from childhood through adulthood.
🔬 BMJ Paediatrics Open
A 2019 review study found that children with Rett syndrome may show subtle signs of the condition before the well-known regression at age 2, suggesting there may be an earlier window for diagnosis and support.
🔬 Neuroscience and Biobehavioral Reviews
This completed study investigated how factors like family functioning, coping methods, and perceived illness burden impact caregivers of individuals with Rett Syndrome.
🔬 National Human Genome Research Institute (NHGRI)
This research digest is for educational and community support purposes only. It is not medical advice, diagnosis, or treatment. Always speak with a qualified doctor, therapist, or medical professional before making decisions about care.
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